Date: 2014-06-13
Type of information: Presentation of results at a congress
phase: 3
Announcement: presentation of results at the European Hematology Association Annual Congress in Milan, Italy
Company: GSK (UK) Ligand Pharmaceuticals (USA - CA)
Product: eltrombopag (Promacta™/Revolade™)
Action
mechanism: thrombopoietin agonist. Eltrombopag works by helping to induce proliferation and differentiation of bone marrow stem cells to increase production of blood cells.
Disease: chronic immune (idiopathic) thrombocytopenic purpura (cITP)
Therapeutic area: Autoimmune diseases - Rare diseases
Country: USA, Argentina, Czech Republic, Germany, Hong Kong, Israel, Italy, Poland, Russian Federation, Spain, Taiwan, Thailand, United Kingdom
Trial
details: PETIT2 was 2-part, double-blind, randomized placebo-controlled and open-label study to investigate the efficacy, safety and tolerability of eltrombopag in pediatric patients with previously treated cITP. The multi-center study enrolled 93 subjects at 38 centers in 14 countries.
The primary objective of the study was to assess the efficacy of eltrombopag, relative to placebo, in achieving platelet counts of =50 Gi/L among pediatric patients with previously treated cITP for at least 12 months. The initial phase of the study compared eltrombopag to placebo for 13 weeks. All study participants were then treated with eltrombopag in the second phase of the study (through to week 24). (NCT01520909)
Latest
news: * On June 16, 2014, Ligand Pharmaceuticals announced that its partner,GSK, presented the results from the Phase 3 PETIT2 study evaluating the efficacy of eltrombopag vs. placebo in pediatric patients with chronic immune (idiopathic) thrombocytopenic purpura (cITP). Eltrombopag met its primary endpoint, achieving a statistically significant improvement in platelet counts with almost 40 percent of patients treated with eltrombopag attaining a consistent platelet response for 6 of 8 weeks compared to placebo (39.7 percent vs. 3.4 percent, respectively, p<0.001). The PETIT2 study results were highlighted as part of a Press Briefing and Oral Presentation at the European Hematology Association Annual Congress in Milan, Italy. Efficacy results for PETIT2 were consistent across age cohorts. The safety profile was consistent with the established profile for eltrombopag and no new safety concerns were observed. The most common adverse events (AEs) occurring most frequently in the eltrombopag arm included nasopharyngitis, rhinitis, cough and respiratory tract infection. Grade 3/4 AEs occurred in 12.7 percent of patients treated with eltrombopag and 10.3 percent of patients in the placebo group. Serious AEs were reported in 8 percent of eltrombopag-treated patients vs. 14 percent in the placebo arm.
GSK also announced that it is moving forward with planned regulatory submissions for a pediatric indication in cITP later this year.
